The U.S. FDA has approved Zycubo (copper histidinate) as the first treatment for Menkes disease, a rare, genetic disease affecting children who cannot absorb copper through their intestines, leading to seizures, weak muscles, a failure to thrive and, ultimately, if left untreated, an early death by age 3.
Biopharma happenings, including deals and partnerships, and other news in brief: Alloy, BCM, Domain, Eisai, Formosa, Johnson & Johnson, Link Cell, Lyra, Metagenomi, Nuvation Bio, Radius Health, Samil, Swiss Rockets, Vaximm.
Regulatory snapshots, including global drug submissions and approvals, and other regulatory decisions and designations: Alessa, Aquestive, Kezar, PDS, Uniqure.
Biopharma happenings, including deals and partnerships, and other news in brief: Acepodia, Anaptys, Avista, Biocytogen, Cytoreason, Dewpoint, GSK, Iktos, Link Cell Therapies, Madrigal, Medilink, Nona, Pfizer, Pierre, Roche, Sanofi, Tesaro.
A second complete response letter issued by the U.S. FDA for Atara Biotherapeutics Inc.’s allogeneic T-cell immunotherapy Ebvallo (tabelecleucel) for Epstein-Barr virus positive post-transplant lymphoproliferative disease is “a complete reversal of position” by the agency, which had previously confirmed the single arm Allele trial was enough to support a BLA under the accelerated approval pathway, the company said.
After raising AU$29 million (US$19.44 million) in a series A round, Rage Biotech Pty Ltd. is beginning phase I trials of its lead candidate, RB-042, an inhaled splice-switching oligonucleotide for treating chronic obstructive pulmonary disease (COPD) and other inflammatory lung diseases.
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